| Size | Price | Stock | Qty |
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| 1mg |
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| Other Sizes |
| Targets |
Golodirsen sodium targets exon 53 of dystrophin pre-mRNA. As a phosphorodiamidate morpholino oligomer (PMO), it binds to the pre-mRNA and modifies the splicing process, leading to exon skipping. This restores the reading frame of the Duchenne muscular dystrophy (DMD) gene, allowing production of a partially functional dystrophin protein. It is used for research of Duchenne muscular dystrophy (DMD).
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| ln Vitro |
In vitro studies demonstrate that Golodirsen sodium is a PMO that specifically targets exon 53 of dystrophin pre-mRNA. It modifies the splicing process to skip exon 53 and restore the reading frame of the DMD gene. This allows production of a partially functional dystrophin protein. Detailed in vitro activity data, including exon skipping efficiency and dystrophin protein restoration, are not extensively documented in the available literature.
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| ln Vivo |
In vivo activity of Golodirsen sodium has been demonstrated in preclinical studies. The compound exhibits consistent pharmacokinetic characteristics in multiple species with plasma half-lives ranging from 2.1 to 8.7 hours. Extensive biodistribution is observed in target skeletal muscle tissues and the kidney. Golodirsen sodium restores the reading frame of the DMD gene by modifying pre-mRNA splicing and skipping exon 53. It is used for research of Duchenne muscular dystrophy.
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| Enzyme Assay |
The in vitro enzyme/receptor binding assay for Golodirsen sodium is not applicable, as the compound is an antisense oligonucleotide that functions through base-pairing with pre-mRNA rather than enzyme inhibition. However, binding assays may be performed to assess its hybridization to target RNA sequences. Standard protocols include electrophoretic mobility shift assays or surface plasmon resonance to measure binding affinity to the exon 53 target sequence.
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| Cell Assay |
In vitro cell-based assays for Golodirsen sodium are conducted using muscle cell lines or patient-derived cells with DMD mutations amenable to exon 53 skipping. Cells are treated with the compound at various concentrations, and exon skipping efficiency is assessed by RT-PCR. Dystrophin protein restoration is measured by Western blot or immunofluorescence. Cytotoxicity is evaluated using standard cell viability assays.
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| Animal Protocol |
In vivo animal studies for Golodirsen sodium have been conducted in mice, rats, and cynomolgus monkeys at doses ranging from 12-960 mg/kg (mice), 100-900 mg/kg (rats), and 5-320 mg/kg (cynomolgus monkeys) via intravenous single dose administration. Pharmacokinetic characteristics and biodistribution were evaluated. Extensive biodistribution was observed in target skeletal muscle tissues and the kidney.
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| ADME/Pharmacokinetics |
Pharmacokinetic properties of Golodirsen sodium include consistent pharmacokinetic characteristics in multiple species with plasma half-lives ranging from 2.1 to 8.7 hours. Extensive biodistribution is observed in target skeletal muscle tissues and the kidney. Doses studied in animals range from 12-960 mg/kg (mice), 100-900 mg/kg (rats), and 5-320 mg/kg (cynomolgus monkeys) via intravenous single dose administration.
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| Toxicity/Toxicokinetics |
Toxicity information for Golodirsen sodium is limited in the search results. As a research-use compound, standard safety precautions for handling should be followed. The compound is designated for research use only and is not for human therapeutic applications. No detailed toxicity data are available from the search results.
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| References | |
| Additional Infomation |
Golodirsen sodium (SRP-4053 sodium) is a PMO that targets exon 53 of dystrophin pre-mRNA for Duchenne muscular dystrophy research. It restores the reading frame by modifying splicing and skipping exon 53. It exhibits consistent PK in multiple species with half-lives of 2.1-8.7 h and biodistribution in skeletal muscle and kidney. It is intended for research use only.
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| Molecular Formula |
C305H481N138NA25O112P25
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| Molecular Weight |
9222.01
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| Appearance |
White to off-white solid powder
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| HS Tariff Code |
2934.99.9001
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| Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month Note: Please store this product in a sealed and protected environment (e.g. under nitrogen), avoid exposure to moisture. |
| Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
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| Solubility (In Vitro) |
Typically soluble in DMSO (e.g. 10 mM)
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| Solubility (In Vivo) |
Note: Listed below are some common formulations that may be used to formulate products with low water solubility (e.g. < 1 mg/mL), you may test these formulations using a minute amount of products to avoid loss of samples.
Injection Formulations
Injection Formulation 1: DMSO : Tween 80： Saline = 10 : 5 : 85 (i.e. 100 μL DMSO stock solution → 50 μL Tween 80 → 850 μL Saline)(e.g. IP/IV/IM/SC) *Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH ₂ O to obtain a clear solution. Injection Formulation 2: DMSO : PEG300 ：Tween 80 : Saline = 10 : 40 : 5 : 45 (i.e. 100 μL DMSO → 400 μLPEG300 → 50 μL Tween 80 → 450 μL Saline) Injection Formulation 3: DMSO : Corn oil = 10 : 90 (i.e. 100 μL DMSO → 900 μL Corn oil) Example: Take the Injection Formulation 3 (DMSO : Corn oil = 10 : 90) as an example, if 1 mL of 2.5 mg/mL working solution is to be prepared, you can take 100 μL 25 mg/mL DMSO stock solution and add to 900 μL corn oil, mix well to obtain a clear or suspension solution (2.5 mg/mL, ready for use in animals). View More
Injection Formulation 4: DMSO : 20% SBE-β-CD in saline = 10 : 90 [i.e. 100 μL DMSO → 900 μL (20% SBE-β-CD in saline)] Oral Formulations
Oral Formulation 1: Suspend in 0.5% CMC Na (carboxymethylcellulose sodium) Oral Formulation 2: Suspend in 0.5% Carboxymethyl cellulose Example: Take the Oral Formulation 1 (Suspend in 0.5% CMC Na) as an example, if 100 mL of 2.5 mg/mL working solution is to be prepared, you can first prepare 0.5% CMC Na solution by measuring 0.5 g CMC Na and dissolve it in 100 mL ddH2O to obtain a clear solution; then add 250 mg of the product to 100 mL 0.5% CMC Na solution, to make the suspension solution (2.5 mg/mL, ready for use in animals). View More
Oral Formulation 3: Dissolved in PEG400 (Please use freshly prepared in vivo formulations for optimal results.) |
| Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
| 1 mM | 0.1084 mL | 0.5422 mL | 1.0844 mL | |
| 5 mM | 0.0217 mL | 0.1084 mL | 0.2169 mL | |
| 10 mM | 0.0108 mL | 0.0542 mL | 0.1084 mL |
*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.
Calculation results
Working concentration： mg/mL；
Method for preparing DMSO stock solution： mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.
Method for preparing in vivo formulation:：Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O，mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
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